RPGRIP1 GENE THERAPY FOR LEBER CONGENITAL AMAUROSIS

摘要

This invention relates to methods for treating subjects with vision loss due to advanced Leber Congenital Amaurosis (LCA), e.g., LCA6, which is due to loss-of-function mutations in the gene encoding the retinitis pigmentosa GTPase regulator interacting-protein-1 (RPGRIP1) protein.